-Patient 1 of Cohort 1 completed the 24-month follow-up timepoint, and at the 24-month post-treatment timepoint Patient 1 continued to experience the disease-modifying effects of BB-301, with deepening improvements in post-swallow residue and total dysphagic symptom burden as compared to the 12-month follow-up timepoint-
- An update on the Interim clinical results for Cohort 2 is planned for mid-2026-
-FDA meeting to formalize the pivotal BB-301 study design expected mid-year-
HAYWARD, Calif.,, Feb. 12, 2026 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ:BNTC) ("Benitec" or the "Company"), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary "Silence and Replace" DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its second fiscal quarter ended December 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission.
"We continue to be encouraged by the benign safety profile and the durability of efficacy demonstrated in our BB-301 clinical development program," said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. "We look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the BB-301 pivotal study design and continuing to present interim clinical results at future medical conferences. I want to sincerely thank our investigators, our clinical advisors, and—most importantly—the patients and families who have made this progress possible."
The recent and upcoming key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia, are outlined below:
Responder Analysis for Study Completers
A Responder Analysis was developed to facilitate standardized evaluation of BB-301 efficacy for each Patient. The Responder Analysis consists of multiple discrete response categories that collectively assess the dysphagic symptom burden in patients with OPMD.
These response categories include:
Patient-Reported Outcome: Patient-reported oral-pharyngeal dysphagia as assessed by the Sydney Swallow Questionnaire (SSQ) total score
Videofluoroscopic Swallowing Study (VFSS) Assessments:
Pharyngeal constrictor muscle function as estimated by the Pharyngeal Area at Maximum Constriction (PhAMPC)
Swallowing efficiency as measured by NRRSv and Total Pharyngeal Residue %(C2-4)2
Frequency of pathologic sequential swallows (SEQ)
Functional Swallowing Capacity: Cold-Water Timed Drinking Test (CWDT)
The evaluation is completed as follows:
Following completion of the 12-month post-treatment follow-up timepoint, each discrete response category is evaluated for each study Completer using prespecified statistical criteria
Results of the statistical characterization of each response category are combined into a single scoring framework that facilitates the overall assessment of clinical benefit achieved by each Completer following treatment with BB-301
A total Score of 5 is possible
Responder status for each Completer will be assigned based on the achievement of statistical criteria for at least 2 out of 5 discrete response categories (≥40%)
Responder Analysis for Study Completers:
All four Cohort 1 Completers were formal responders to BB-301, demonstrating durable response to BB-301 at the conclusion of the 12-month statistical follow-up period.
24-Month Post-Treatment Follow-Up for Patient 1 of Cohort 1
At the 24-month post-BB-301 treatment follow-up timepoint, Patient 1 of Cohort 1 continued to demonstrate robust, disease-modifying outcomes. Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and as compared to the 12-month post-treatment follow-up timepoint as assessed by VFSS. Patient 1 also experienced deepening improvements in total dysphagic symptom burden as assessed by the SSQ.
Enrollment into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing:
The first Patient in Cohort 2 was safely treated with the higher-dose of BB-301 in 4Q of 2025 and an update on the interim clinical results of Cohort 2 is planned for mid-2026.
Corporate Updates:
The Company plans to engage with the FDA in mid-2026 to confirm the BB-301 pivotal study design. In November 2025, Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and the proprietary Responder Analysis planned for use in the BB-301 pivotal study. Previously, BB-301 has received Orphan Drug Designation from the EMA and the FDA.
Financial Highlights
Second Quarter 2026 Financial Results
Total Expenses for the quarter ended December 31, 2025 were $13.4 million compared to $10.8 million for the quarter ended December 31, 2024. The Company incurred $5.8 million of research and development expenses which was in line with $5.4 million for the comparable quarter ended December 31, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative expenses were $7.5 million compared to $5.4 million for the quarter ended December 31, 2024.
The loss from operations for the quarter ended December 31, 2025, was $13.4 million compared to a loss of $10.8 million for the quarter ended December 31, 2024. Net loss attributable to shareholders for the quarter ended December 31, 2025, was $11.8 million, or $(0.26) per basic and diluted share, compared to a net loss of $9.6 million, or $(0.26) per basic and diluted share for the quarter ended December 31, 2024. As of December 31, 2025, the Company had $189 million in cash and cash equivalents, which includes $0.1 million from the exercise of warrants during the six-month period ended December 31, 2025.
BENITEC BIOPHARMA INC.Consolidated Balance Sheets(in thousands, except par value and share amounts)
December 31,2025
June 30,2025
(Unaudited)
Assets
Current assets:
Cash and cash equivalents
$
188,790
$
97,744
Restricted cash
113
113
Trade and other receivables
130
33
Prepaid and other assets
562
628
Total current assets
189,595
98,518
Property and equipment, net
115
131
Deposits
55
55
Prepaid and other assets
11
28
Right-of-use assets
905
860
Total assets
$
190,681
$
99,592
Liabilities and stockholders' equity
Current liabilities:
Trade and other payables
$
1,850
$
1,022
Accrued employee benefits
483
426
Lease liabilities, current portion
468
354
Total current liabilities
2,801
